NAHD is an Indo-Japan collaborative project supported by the DBT, Ministry of Science and Technology, Government of India. NAHD is part of the “Accelerating the application of Stem cell technology in Human Disease” (ASHD) project, to exploit cutting-edge research in human genetics, stem cell technology, and clinical investigations to provide translational gene and cell therapy solutions for patients with hereditary blood disorders. The major components of the NAHD program are Gene Therapy, Haplobanking and Control Program of Thalassemia and Sickle Cell Anemia (SCD).

Gene Therapy

1. To develop a clinical trial for the treatment of Haemophilia B by exploring the use of vectors using AAV (Adeno-associated virus) serotypes. More specifically, an AAV-3 based vector construct with the transgene Factor IX cassette has been designed and codon-optimized and its efficacy are being tested in humanized mice and non-human primate models. Regulatory approvals are required to transfer the gene therapy product for GMP grade industrial manufacture and Haemophilia B clinical trials in India.

2. To develop novel preclinical methods/technologies such as lentiviral-based recombinant vectors, genome editing tools and iPSC (induced Pluripotent Stem Cell)-based disease modeling approaches to effectively treat hereditary blood disorders.

Applications of Induced Pluripotent Stem Cell (iPSC) Technology: Haplobanking

Haplobanking component aims to create a large iPSC repository from the blood of HLA-homozygous haplotype individuals (top 10 HLA haplotypes represented in the Indian population). This bank of cells is intended to be the starting material for reprogramming and generation of human iPSCs. Human iPSCs could be used as a cell therapy option for treating a variety of diseases including but not limited to haematological diseases.

Control of Thalassemia & Sickle Cell Disease program for Odisha

Control Program of Thalassemia and SCD is focussed on constructing a model for a community outreach program that serves to spread public awareness of blood disorders (Thalassemia and Sickle cell disease) and to encourage genetic counseling and carrier status testing, thereby preventing the birth with thalassemia major.